Neurological

Novel CRISPR therapy receives orphan drug status for transthyretin amyloidosis

The Food and Drug Administration (FDA) granted NTLA-2001 orphan drug status for the treatment of transthyretin (ATTR) amyloidosis.

ATTR amyloidosis is a rare, progressive, life-threatening disease caused by mutations in the transthyretin (TTR) gene that result in the production of a structurally abnormal TTR protein that is prone to misfolding. The damaged proteins build up in the body as deposits of amyloid and cause complications in several tissues, including the heart, nerves, and digestive system.

Using CRISPR / Cas9 technology, investigative therapy was developed to manipulate disease-causing genes in the human body. Given as a single intravenous infusion, NTLA-2001 inactivates the TTR gene in liver cells, preventing the production of misfolded TTR protein.

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The company is currently investigating the efficacy and safety of NTLA-2001 in adults with hereditary ATTR amyloidosis with polyneuropathy in a Phase 1 clinical trial (ClinicalTrials.gov Identifier: NCT04601051). Interim results showed that treatment with NTLA-2001 resulted in a mean reduction in serum TTR of 87% and a maximum reduction in serum TTR of 96% by day 28 in 6 patients.

FDA orphan drug status is granted to drugs that are intended to treat or prevent rare diseases or disorders that affect fewer than 200,000 people.

“Orphan drug status underscores the FDA’s recognition of the potential of NTLA-2001 as a novel single-dose therapy for the treatment of ATTR amyloidosis,” said John Leonard, MD, president and chief executive officer of Intellia.

References

  1. Intellia Therapeutics Receives FDA Orphan Drug Status for NTLA-2001, an investigational CRISPR therapy for the treatment of transthyretin (ATTR) amyloidosis. Press release. Intellia Therapeutics, Inc. Accessed October 22, 2021. https://www.globenewswire.com/news-release/2021/10/21/2318228/0/en/Intellia-Therapeutics-Receives-US-FDA-Orphan- Drug-name-for-NTLA-2001-an-investigation-CRISPR-therapy-for-the-treatment-of-transthyretin-ATTR-amyloidosis.html.
  2. JD Gillmore, E. Gane, J. Taubel et al. CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis. N Engl J Med. Published online August 5, 2021. doi: 10.1056 / NEJMoa2107454

This article originally appeared on MPR

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